Transcriptomic-guided compound prioritization and proteomics validation for HNRNPU deficiency identify signalling correction
Xuan Ye, Diana Tikhomirova, Marika Oksanen, Massimiliano Gaetani, Hassan Gharibi, Francesca Mastropasqua, Kristiina Tammimies
The paper develops a transcriptomic-guided compound prioritization pipeline and validates it with proteomics, identifying two compounds that partially rescue HNRNPU deficiency phenotypes.
bioRxiv · May 7, 2026 · PreprintMolecular Biology